title: Autologous stem cell transplantation with PCR-negative graft would be associated with a favorable outcome in core-binding factor acute myeloid leukemia.
abstract: Although core-binding factor acute myeloid leukemia (CBF-AML) is generally considered to be a low-risk form of AML, the survival rate is still 50% to 60%. To evaluate the effectiveness of autologous stem cell transplantation (ASCT) with a PCR-negative graft we analyzed a series of consecutive CBF-AML patients. Between 1997 and 2006, 18 patients aged<60 years were referred under a diagnosis of CBF-AML. Peripheral blood stem cells (PBSC) were collected after a second or further course of postremission therapy. When >2.0x10(6)/kg CD34-positive cells with minimal residual disease (MRD) undetectable by nested polymerase chain reaction (PCR) had been collected, ASCT was performed with busulfan, etoposide, and cytarabine combined with granulocyte colony-stimulating factor. Event-free survival (EFS) and complications of ASCT were then assessed. Fourteen of the 18 patients received ASCT. The median observation period was 4.4 years. The 5-year EFS was 93% for ASCT patients, despite the presence of adverse factors. In 8 of 10 patients who had detectable MRD in the bone marrow before ASCT, MRD became undetectable after ASCT. Neutrophils recovered promptly within 2 weeks, but platelets recovered relatively slowly. Half of the patients suffered from varicella zoster virus infection. Although 1 case of myelodysplastic syndrome occurred, there was no case of relapse. ASCT with a PCR-negative graft was associated with excellent EFS. For patients with CBF-AML, especially with adverse factors or remnant MRD in the bone marrow, this strategy is the treatment of choice.
authors.affiliation: Division of Hematology, Kanto Medical Center NTT EC, Department of Internal Medicine, Nippon Medical School, Tokyo, Japan. nakasone-tky@umin.ac.jp
authors.forename: Hideki
authors.initials: H
authors.lastname: Nakasone
authors.forename: Koji
authors.initials: K
authors.lastname: Izutsu
authors.forename: Satoshi
authors.initials: S
authors.lastname: Wakita
authors.forename: Hiroki
authors.initials: H
authors.lastname: Yamaguchi
authors.forename: Michiko
authors.initials: M
authors.lastname: Muramatsu-Kida
authors.forename: Kensuke
authors.initials: K
authors.lastname: Usuki
mesh_terms: D000328:Adult; D000368:Aged; D050660:Core Binding Factors; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D008991:Monitoring, Physiologic / Q000379:methods; D018365:Neoplasm, Residual; D036102:Peripheral Blood Stem Cell Transplantation; D016133:Polymerase Chain Reaction; D020127:Recovery of Function; D012189:Retrospective Studies; D015996:Survival Rate; D014182:Transplantation, Autologous
chemical_list: D050660:Core Binding Factors